Company (location)







AB Science SA, of Paris


Tyrosine kinase inhibitor targeting mast cells and macrophages

Nonresectable or metastatic stage 3 or stage 4 melanoma

Study in patients whose disease carries mutation in the juxta-membrane domain of c-Kit showed objective response rate of 39.1% (p=0.0012) in first-line treatment and 33.3% (p=0.0049) regardless of line of treatment; there were 2 complete responses, including 2 complete response lasting for 1,030 days


Abbvie Inc., of North Chicago

Rovalpituzumab tesirine (Rova-T)

Antibody-drug conjugate targeting delta-like protein 3

Advanced small-cell lung cancer

During a pre-planned interim analysis of the Meru study, Rova-T produced no survival benefit compared to placebo; study closed on recommendation of the independent data monitoring committee; Rova-T research and development program has been terminated


Amgen Inc., of Thousand Oaks, Calif.; Allergan plc, of Dublin


Biosimilar candidate to Rituxan (rituximab, Roche Holding AG / Biogen Inc.)

CD20-positive B-cell non-Hodgkin's lymphoma

Primary endpoint, an assessment of overall response rate by week 28, was within the prespecified margin for ABP-798 compared to Rituxan, showing clinical equivalence; safety and immunogenicity were comparable


Astrazeneca plc, of Cambridge, U.K.

Tagrisso (osimertinib)

Tyrosine kinase inhibitor targeting EGFR

Locally advanced or metastatic non-small-cell lung cancer

Overall survival results from Flaura trial in patients whose tumors have EGFR mutations showed statistically significant and clinically meaningful improvement in OS, a secondary endpoint, compared with erlotinib or gefitinib, both of which were previous standard of care


Astrazeneca plc, of Cambridge, U.K.

Imfinzi (durvalumab) + tremelimumab

PD-L1 checkpoint inhibitor + anti-CTLA4 antibody

Non-small-cell lung cancer

Neptune trial evaluating combination vs. standard-of-care platinum-based chemotherapy in previously untreated metastatic disease did not meet primary endpoint of improving overall survival in primary analysis population of individuals with blood tumor mutational burden of 20 or more mutations per megabase


Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J.

Lynparza (olaparib)

PARP inhibitor

Metastatic castration-resistant prostate cancer with BRCA1/2 or ATM gene mutations and progression on new hormonal anticancer treatments

Study met primary endpoint with an improvement in radiographic progression-free survival for Lynparza compared to enzalutamide or abiraterone; data to be presented at an upcoming medical meeting


Astrazeneca, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J.

Lynparza (olaparib)

PARP inhibitor

First-line maintenance of advanced ovarian cancer

The Paola-1 study met its primary endpoint with Lynparza plus Avastin (bevacizumab, Roche Holding AG) improving progression-free survival compared to Avastin alone; data to be presented at an upcoming medical meeting


Athenex Inc., of Buffalo, N.Y.

Oral paclitaxel


Metastatic breast cancer

In the 402-patient study, oral paclitaxel produced an overall response rate of 36% compared to 24% for intravenous (I.V.) paclitaxel (p=0.01); at data cutoff, progression-free survival (p=0.077) and overall survival (p=0.11) were better for oral paclitaxel over I.V. paclitaxel


Deciphera Pharmaceuticals Inc., of Waltham, Mass.


KIT and PDGFR-alpha kinase switch control inhibitor

Gastrointestinal stromal tumor

Trial achieved primary endpoint, with ripretinib having significantly improved progression-free survival vs. placebo in patients with fourth-line and fourth-line or greater GIST; expects to file NDA in first quarter of 2020


Mologen AG, of Berlin

Lefitolimod (MGN-1703)

TLR9 agonist

Metastatic colorectal cancer

Top-line data from Impala study testing maintenance therapy in patients presenting with an objective tumor response following first-line induction therapy missed the primary endpoint of overall survival, with median OS of 22 months vs. 21.9 months for lefitolimod vs. control group (p=0.2765); timepoint-related OS and predefined subgroup analyses also did not indicate benefit, while standard of care was superior to lefitolimod on progression-free survival


Progenics Pharmaceuticals Inc., of New York

Pyl (18F-DCFPyl)

PSMA-targeted small-molecule PET/CT imaging agent

Prostate Cancer

Completed enrollment ahead of schedule in Condor study to test diagnostic performance and clinical impact in men with biochemical recurrence of prostate cancer


Rafael Pharmaceuticals Inc., of Cranbury, N.J.

CPI-613 (devimistat)

Targets mitochondrial tricarboxylic acid cycle

Relapsed or refractory acute myeloid leukemia

Expanded Armada 2000 study into Austria, South Korea and Spain; pivotal study is testing devimistat in combination with high-dose cytarabine and mitoxantrone in older patients


Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab)

PD-L1-inhibiting antibody

Locally advanced or metastatic urothelial carcinoma

Imvigor130 study met co-primary endpoint of investigator-assessed progression-free survival; combination with platinum-based chemotherapy showed statistically significant PFS in patients with previously untreated disease vs. chemotherapy alone; overall survival data deemed encouraging though not yet mature


Sesen Bio Inc., of Cambridge, Mass.


Antibody-drug conjugate binding to EpCAM

Non-muscle invasive bladder cancer

Updated data from Vista study further support drug in patients with high-risk, bacillus Calmette-Guerin unresponsive disease; 90% of all 133 treated patients estimated to remain progression-free for 2 years or longer, using Kaplan-Meier method; 29% estimated to remain event-free at 12 months; 96% estimated to have overall survival of 2 years or longer; BLA submission expected to be initiated in the fourth quarter of 2019


Soligenix Inc., of Princeton, N.J.

SGX-942 (dusquetide)

Innate defense regulator in a new class of short, synthetic peptides

Oral mucositis in patients with head and neck cancer

Data monitoring committee recommended that approximately 70 additional subjects be randomized into the trial, increasing the study sample size from 190 to 260 evaluable subjects


Transgene SA, of Strasbourg, France

Pexa-Vec (JX-594)

Oncolytic virus

Liver cancer

Independent data monitoring committee of Phocus study completed planned interim analysis and recommended enrollment be stopped, as study is unlikely to meet primary objective



Astrazeneca, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Heart failure with reduced ejection fraction

In the DAPA-HF study, Farxiga improved the composite endpoint of cardiovascular death or the worsening of heart failure (defined as hospitalization or an urgent heart failure visit), compared to placebo; data to be presented at an upcoming medical meeting



Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del.

Olumiant (baricitinib)

JAK inhibitor

Moderate to severe atopic dermatitis

In the Breeze-AD7 study, 30.6% of patients taking 4 mg baricitinib plus standard-of-care topical corticosteroids had a validated Investigator's Global Assessment for AD score of clear or almost clear skin at week 16 compared to 14.7% of patients on placebo plus corticosteroids (p≤0.01); 47.7% of patients on drug reached EASI75 compared to 22.9% for placebo (p≤0.001); 44.0% of patients on drug had a 4-point improvement in Itch NRS compared to 20.2% of patients taking placebo (p≤0.001)


MC2 Therapeutics A/S, of Copenhagen

MC2-01 PAD cream (calcipotriene and betamethasone dipropionate)

Calcitriol receptor agonist/steroid receptor agonist

Plaque psoriasis

Completed enrollment for EU study comparing product to Daivobet gel


Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.

Dupixent (dupilumab)

IL-4/IL-13-targeting monoclonal antibody

Atopic dermatitis

Top-line data from pivotal study in pediatric population, ages 6 to 11, showed adding Dupixent to standard-of-care topical corticosteroids (TCS) significantly improved measures of overall disease severity, skin clearing, itching and health-related quality of life, compared to TCS alone; at 16 weeks, 33% who received Dupixent every 4 weeks (300 mg) and 30% who received Dupixent every 2 weeks (100 mg or 200 mg) achieved clear or almost clear skin, compared to 11% for placebo (p<0.0001 and p=0.0004, respectively)



Akcea Therapeutics Inc., of Boston, an affiliate of Ionis Pharmaceuticals Inc.

Waylivra (volanesorsen)

Reduces production of apolipoprotein C-III

Familial chylomicronemia syndrome

Final results from Approach study published in The New England Journal of Medicine show treated patients achieved statistically significant mean reduction in triglycerides of 77% from baseline (mean reduction of 94% vs. placebo) at 3 months; levels of apoC-III also decreased by an average of 84% from baseline at 3 months (p<0.001)


Lexicon Pharmaceuticals Inc., of The Woodlands, Texas

Zynquista (sotagliflozin)

Dual inhibitor of SGLT1 and SGLT2

Type 1 diabetes

52-week cardiorenal results from a pooled analysis of Tandem1 and Tandem2 studies, published in Diabetes Care, demonstrated changes in clinical biomarkers such as estimated glomerular filtration rate (eGFR), hematocrit, serum albumin, uric acid, systolic blood pressure and urinary albumin-to-creatinine ratio that suggest Zynquista may reduce cardiovascular risk and progression of chronic kidney disease; treatment was associated with short- and long-term renal hemodynamic changes; after cessation of 52 weeks of therapy, eGFR was comparable to baseline and significantly higher than placebo in Zynquista-treated patients


Opko Healthy Inc., of Miami

Somatrogon (hGH-CTP)

Human growth hormone with a carboxyl terminal peptide

Growth hormone deficiency

Last patient completed the final visit in the 224-patient study comparing once-weekly somatrogon to once-daily Genotropin (somatropin, Pfizer Inc.) with a primary endpoint of height velocity at 52 weeks; top-line data expected in the 4Q19


Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.


Monoclonal antibody targeting angiopoietin-like 3

Homozygous familial hypercholesterolemia

After 24 weeks of treatment, evinacumab reduced low-density lipoprotein (LDL) cholesterol by 47% compared to a 2% increase for placebo (p<0.0001); 47% of patients treated with evinacumab achieved LDL cholesterol levels less than 100 mg/dL, compared to 23% of placebo-treated patients (nominal p=0.0203)


Rhythm Pharmaceuticals Inc., of Boston


Melanocortin-4 receptor agonist

Pro-opiomelanocortin deficiency obesity

8 of 10 patients achieved weight loss of at least 10% (p<0.0001); mean reduction in body weight was 25.4% (p<0.0001); mean reduction in most hunger rating was 27.8% (p=0.0005); 50% of patients had at least a 25% improvement in self-reported hunger scores (p=0.0004)


Rhythm Pharmaceuticals Inc., of Boston


Melanocortin-4 receptor agonist

Leptin receptor deficiency obesity

5 of 11 patients achieved weight loss of at least 10% (p<0.0001); mean reduction in body weight was 12.5% (p<0.0001); mean reduction in most hunger rating was 41.9% (p=0.0001); 72.7% of patients had at least a 25% improvement in self-reported hunger scores (p=0.0001)


The Medicines Co., of Parsippany, N.J.


siRNA targeting PCSK9

Atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents with elevated low-density lipoprotein cholesterol

The Orion-11 study met all of its primary and secondary endpoints; data to be presented at the European Society of Cardiology's ESC Congress 2019



Atlantic Healthcare plc, of Cambridge, U.K.

Camligo (alicaforsen enema) 

Alicaforsen enema 


Using an adaptation of the Mayo Score of improvement in endoscopic remission and bowel frequency, the trial did not meet its co-primary endpoints


Innovate Biopharma-ceuticals Inc., of Raleigh, N.C.

INN-202 (larazotide acetate)

Tight junction regulator

Celiac disease

Dosed the first patient in the CeD LA 3001 trial; about 600 subjects to be enrolled; primary objective is to test drug as an adjunct therapy in those who still experience symptoms despite a gluten-free diet


Genitourinary/Sexual Health

Astellas Pharma Inc., of Tokyo


Oral, nonhormonal selective neurokinin-3 receptor antagonist

Moderate to severe vasomotor symptoms associated with menopause

Dosed first patient in Skylight 1 pivotal trial to evaluate 30-mg and 45-mg once-daily treatment in reducing VMS frequency and severity


Evofem Biosciences Inc., of San Diego


Vaginal Ph regulator


Additional data from Ampower trial showed Amphora might improve sexual satisfaction and have a positive impact on women's sex lives, while also confirming previously reported safety and efficacy data


Mallinckrodt plc, of Staines-upon-Thames, U.K.


Vasoactive drug

Hepatorenal syndrome type 1

Pivotal Confirm study met primary endpoint of verified HRS-1 reversal (p=0.012), which includes renal function improvement, avoidance of dialysis and short-term survival; NDA expected in 2020


Myovant Sciences Inc., of Brisbane, Calif.


Gonadotropin-releasing hormone receptor antagonist

Endometriosis-associated pain

Completed enrollment of about 600 patients in the SPIRIT 2 study; data expected in the first quarter of 2020


Vascular Therapies Inc., of Cresskill, N.J.


Formulation for intraoperative local, perivascular drug delivery

Kidney disease

Completed enrollment in U.S. Access trial; testing formulation to improve outcomes in patients undergoing surgical creation of an arteriovenous fistula to provide vascular access for hemodialysis; primary endpoint is Fistula Suitability for Dialysis at 6 months, and results are expected in the second quarter of 2020



Glycomimetics Inc., of Rockville, Md., and Pfizer Inc., of New York

Rivipansel (GMI-1070)

Pan-selectin antagonist

Sickle cell disease

Reset pivotal trial did not meet primary endpoint, defined as readiness-for-discharge for patients, ages 6 and older, who were hospitalized by vaso-occlusive crises and required intravenous opioids; key secondary endpoints, including time-to-discharge, cumulative I.V. opioid consumption and time to discontinuation of I.V. opioids, also were not met



Kedrion Biopharma Inc., of Fort Lee, N.J.

10% intravenous immunoglobulin


Primary immunodeficiency diseases

Enrolled first of about 50 patients in the Cares10 study that is expected to be completed by mid-2021


Novavax Inc., of Gaithersburg, Md.


Aluminum adjuvanted respiratory syncytial virus fusion protein nanoparticle vaccine

RSV infection prophylaxis

New data from the Prepare study showed the vaccine produced a 59.6% reduction in the incidence of serious adverse events diagnosed as pneumonia, with confirmation by chest x-ray, which extended through the first year of life; in x-ray-confirmed pneumonia associated with detection of RSV, efficacy was 72.9% through 180 days of life



Basilea Pharmaceutica Ltd., of Basel, Switzerland


Cephalosporin antibiotic

Acute bacterial skin and skin structure infections

Top-line results showed 679-patient Target study met prespecified primary objective of noninferiority (within the prespecified margin of 10%) to vancomycin plus aztreonam in intent-to-treat (ITT) population; primary endpoint of early clinical response was based on a 20% or more reduction from baseline in lesion size at 48 to 72 hours after start of drug administration; ceftobiprole was also noninferior to vancomycin plus aztreonam for the prespecified secondary endpoint of investigator-assessed clinical success, based on resolution of baseline signs and symptoms of infection, in ITT and clinically evaluable populations at test-of-cure visit, 15 to 22 days after randomization


Novan Inc., of Morrisville, N.C.


Topical gel formulation of berdazimer sodium

Molluscum contagiosum infection

Recently completed patient recruitment in pivotal ongoing B-Simple trial; enrolled about 680 patients with 3 to 70 molluscum lesions at baseline


Venatorx Pharmaceuticals Inc., of Malvern, Pa.


Beta-lactamase inhibitor

Complicated urinary tract infections

Started enrollment in the 582-patient study testing the efficacy, safety and tolerability of VNRX-5133 plus cefepime compared to meropenem using clinical cure and microbiological eradication as the primary composite efficacy endpoint; top-line data expected by the end of 2020


Viiv Healthcare Ltd., of London

Cabotegravir and rilpivirine (Edurant, Johnson & Johnson) combination

HIV-1 integrase strand transfer inhibitor; non-nucleoside reverse transcriptase inhibitor

HIV-1 infection in patients whose viral load is suppressed and who are not resistant to cabotegravir or rilpivirine

Met the primary endpoint, showing that the long-acting regimen of cabotegravir and rilpivirine, injected every two months, was non-inferior to cabotegravir and rilpivirine administered every month at week 48



Astrazeneca plc, of Cambridge, U.K.


Monoclonal antibody targeting subunit 1 of the type I interferon receptor

Systemic lupus erythematosus

The 373-patient Tulip 2 study met its primary endpoint with anifrolumab reducing disease activity compared to placebo, as measured by the British Isles Lupus Assessment Group based Composite Lupus Assessment at week 52; data to be presented at a forthcoming medical meeting


Radius Health Inc., of Waltham, Mass.

Abaloparatide-transdermal patch

PTH type 1 receptor agonist


Randomized first patient in Wearable study enrolling postmenopausal subjects with osteoporosis at high risk of fracture; primary endpoint is percentage change in lumbar spine bone mineral density at 12 months



Aztherapies Inc., of Boston


Combination of 2 previously approved small molecules that have been re-engineered 

Alzheimer's disease, early stage

Target enrollment reached


Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn.


Calcitonin gene-related peptide receptor antagonist


Completed enrollment in the study measuring the mean number of migraine days per month as the primary endpoint


Eli Lilly and Co., of Indianapolis

Emgality (galcanezumab-gnlm)

CGRP-targeting monoclonal antibody


Conquer study in preventive treatment for chronic and episodic migraine in patients with documented previous failures on 2 to 4 different standard-of-care medication categories met primary and all key secondary endpoints; demonstrated superiority vs. placebo in overall mean change from baseline in number of monthly migraine headache days across months 1 through 3, reducing monthly headache days by 4.1 days (p<0.0001) vs. 1 day with placebo in total population


Heron Therapeutics Inc., of San Diego


Local anesthetic bupivacaine plus nonsteroidal anti-inflammatory drug meloxicam

Pain associated with open inguinal hernia repair surgery with mesh

Data from the EPOCH 2 study published in Hernia showed HTX-011 provided superior and sustained pain reduction compared to placebo and bupivacaine solution through the 72-hour postoperative window; a lower proportion of patients reported severe pain through 72 hours on HTX-011 compared to placebo and bupivacaine solution; through 72 hours, opioid consumption was lower and the proportion of opioid-free patients was higher for HTX-011 compared to placebo and bupivacaine solution


Mitsubishi Tanabe Pharma America Inc., Jersey City, N.J.


Continuous subcutaneous carbidopa/levodopa

Parkinson's disease

Started the Boundless study of approximately 300 patients testing the efficacy, safety and tolerability of ND-0612 compared to immediate-release carbidopa/levodopa; primary endpoint is daily "good on time," defined as the sum of on time without dyskinesia and on time with non-troublesome dyskinesia


Retrophin Inc., of San Diego


Small-molecule replacement therapy designed to pass the blood-brain barrier and be converted to phosphopantothenate

Pantothenate kinase-associated neurodegeneration

Missed primary and secondary endpoints as compared to placebo


Satsuma Pharmaceuticals Inc., of San Francisco


Dihydro-ergotamine (DHE) nasal powder


Dosed the first patient in its EMERGE efficacy trial of for the acute treatment of migraine; the study will involve approximately 1,140 migraine patients in the U.S. with participants randomized (1:1:1) to receive 1 of 3 treatments: STS-101 DHE 3.9 mg, STS-101 DHE 5.2 mg or matching placebo and instructed to treat their next migraine attack of at least moderate pain severity with the allocated blinded study medication; top-line data is expected in the second half of 2020


Teva Pharmaceutical Industries Ltd., of Jerusalem

Ajovy (fremanezumab)

CGRP receptor antagonist


Data from Focus study found drug was superior to placebo across all primary and secondary endpoints in 838 adults with episodic or chronic migraine who previously experienced inadequate responses to 2 to 4 classes of preventive treatments; reductions from baseline in monthly days with migraine, moderate to severe headache or use of acute headache medications were about 3.5 days (30 percentage points) greater with fremanezumab than placebo (p<0.0001)


Vistagen Therapeutics Inc., of South San Francisco


Oral NMDA receptor glycine site antagonist

Major depressive disorder

Completed target enrollment of 180 patients in Elevate trial testing AV-101 as adjunctive treatment, together with FDA-approved oral antidepressant (AD), in adults with inadequate therapeutic response to current AD; top-line results expected before year-end



Outlook Therapeutics Inc., of Cranbury, N.J.


Ophthalmic formulation of VEGF antibody bevacizumab

Wet age-related macular degeneration

Completed enrollment of 61 patients in the NORSE 1 study comparing ONS-5010 to Lucentis (ranibizumab, Roche Holding AG/Novartis AG); data expected in the third quarter of 2020



Astrazeneca plc, of London

Breztri Aerosphere (budesonide; glycopyrronium/formoterol fumarate 320/14.4/9.6 mcg)

Glucocorticoid; muscarinic acetylcholine receptor binder/long-acting selective beta2-adrenergic receptor agonist

Chronic obstructive pulmonary disease

Demonstrated a statistically-significant reduction in the rate of moderate or severe exacerbations compared with dual-combination therapies Bevespi Aerosphere (glycopyrronium/formoterol fumarate 14.4/9.6 mcg, Astrazeneca plc) and Astrazeneca's PT-009 (budesonide/formoterol fumarate 320/9.6 mcg)



For more information about individual companies and/or products, see Cortellis.


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